Ascent Therapeutics’ Blood Cancer Drug Could Rival Genzyme’s Mozobil

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Ascent Therapeutics is advancing several of its proprietary “pepducin” drugs through early testing, with a lead candidate emerging as a potential rival to Genzyme’s drug plerixafor (Mozobil) for patients with certain types of blood cancer. Cambridge, MA-based Ascent is touting some of its early research at a biotechnology conference in Zurich, Switzerland this week in hopes of attracting new pharmaceutical partners.

Rick Jones, CEO of Ascent, spoke with me from the Sachs Biotechnology in Europe Conference in Zurich yesterday about the startup’s research progress. Among other developments, the firm is saying that its drug was at least as effective as Genzyme’s plerixafor in spurring the movement of stem cells out of the bone marrow in animal tests. The potential drug could be used like plerixafor for patients with such blood cancers as non-Hodgkin’s lymphoma and multiple myeloma who need blood-forming stem cell transplants.

Ascent has enough cash in the bank from its $19 million Series A round to support its operations for about a year, Jones said. That’s not a long time in biotech, since it often takes a decade or longer to develop and garner approval for a new drug. The CEO wants to form two or more partnerships with large pharma companies not only to advance his company’s research funding and scope, but also to win more confidence from venture backers and attract future rounds of financing. Meantime, Jones is hoping that Swiss drug giant Novartis opts to exercise an option it has purchased from Ascent to gain certain rights to the drug candidates for undisclosed uses.

“With the current environment we just can’t go out and ask for $20 million or $30 million to get ourselves into the clinic without having some other proof that we’ve succeeded in building up this platform,” Jones said. “And the venture capitalists are basically telling me that what they’d like to see is another couple of [pharma] companies buy into the platform.”

In a sense, Ascent is fortunate to have closed its first round of venture capital—from blue chip VC outfits including HealthCare Ventures, Novartis Option Fund, and TVM Capital, no less—before the financial markets collapsed last September. And though Jones declined to say how long Novartis has to exercise its option with Ascent, he noted that it could bring his startup more than $200 million in potential milestone payments.

Aside from financial issues, Ascent appears to have made good on identifying drug candidates by the end of this year, as Jones told me the firm planned to do when it came out of stealth mode last October. He said that in addition to the drug identified for stem cell mobilization, the firm has also engineered one of its proprietary molecules to treat an undisclosed cardiovascular condition and another to treat a gastrointestinal disorder.

So-called “pepducins,” which were discovered in a lab at Tufts Medical Center in the 1990s, are synthesized amino acid chains attached to fatty molecules. The drugs are intended to home in on signaling proteins called G-protein coupled receptors (GPCRs) that span the surfaces of cells and are the main targets for many big moneymakers such as Eli Lilly’s schizophrenia drug olanzapine (Zyprexa) and the heartburn pill ranitidine (Zantac). Ascent’s advantage, Jones said, is its ability to engineer customized pepducins that can target the interior side of the GPCR molecule, while most other drugs are thought to bond to the part that sticks out of the cell’s surface.

Despite its progress, the firm no longer plans to use its first-round financing to launch human clinical trials sometime in 2010, Jones said. Instead, it will build out its technology platform in hopes of attracting partnership deals with drug companies. However, if Novartis decides to exercise its option with Ascent, Jones said the company would be in a position to enter an initial clinical trial by late next year.

Jones sees a lot of potential for the firm’s lead drug for cancer patients beyond stem cell mobilization. (Cambridge-based biotech powerhouse Genzyme (NASDAQ: GENZ) has garnered FDA and European approval for that use of plerixafor in combination with granulocyte-colony stimulating factor.) Jones said that he believes that his firm’s drug candidate could also be used to free cancer cells from the bone marrow into the bloodstream, where they are more easily destroyed by chemotherapy drugs. This would be useful for treating leukemia and other malignancies of the bone marrow.

So, like other platform companies, Ascent has a plethora of potential uses of its science. Yet the key for the company now is to find another big pharma company or two to buy into that potential.